Treating the cause

London – The EU agency recommended Kalydeco (ivacaftor) from the US-biotech Vertex for approval. The treatment targets patients from the age of six years on who have a G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene.

Kalydeco was already approved by the FDA at the end of January this year. According to the European agency, Kalydeco offers an innovative therapeutic approach for patients with cystic fibrosis: it is the first treatment that targets the underlying mechanism of the disease, by restoring the function of the mutated CFTR protein.

Currently available therapies for patients with cystic fibrosis only address the consequences of the disease, not the underlying defect. Clinical studies showed that Kalydeco improved pulmonary function in cystic fibrosis patients with the specific G551D-CFTR mutation. About half of the world's CF patients reside in Europe, where about 35,000 people have the disease. The European Commission will consider the CHMP's recommendation in its final decision on Kalydeco. According to PM live, the European recommendation comes almost four months after Kalydeco was cleared by the US Food and Drug Administration (FDA), with the US agency taking just three months to review Vertex' marketing application. This backs outcomes from a recent report that described how the FDA had a faster approval process than the EMA.

29.07.2015 Newly founded Mereo BioPharma has acquired three mid-stage clinical assets from Novartis. In turn, the Swiss pharma concern will hold a stake in the UK company. Other investors are also on board.

24.07.2015 Biotech companies across Europe continue to raise cash on the public markets, generating €3bn in total financing proceedings in the first half of 2015. Compared to 2014, this is almost twice as much.

23.07.2015 US money keeps flowing into Europe's biotechs. As the race for the peanut allergy market heats up, NASDAQ listed French biotech DBV has announced the successful closing of US$281m (€256m) share offering for its allergy products.

16.07.2015 Sanofi has announced plans to restructure its business in five standalone global business units starting in January 2016. The goal is to promote growth and help the company launch a catalogue of new drugs over the next five years.

15.07.2015 GlaxoSmithKline (GSK) has become the first drugmaker to join the Francis Crick Institute. It is a consortium made up of six scientific and academic institutes to conduct research and drug development in the UK. The new institute is set to become a world-leading centre of biomedical research and innovation.

10.07.2015 Karolinska Development has slashed the reported value of its portfolio by almost half by revising its valuation model for early stage companies and divesting its shares in pain med maker Pharmanest.

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